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Comparative Effectiveness of Anti-TNF in Combination with Low Dose Methotrexate vs Anti-TNF Monotherapy in Pediatric Crohn’s Disease: a Pragmatic Randomized Trial
Journal article   Peer reviewed

Comparative Effectiveness of Anti-TNF in Combination with Low Dose Methotrexate vs Anti-TNF Monotherapy in Pediatric Crohn’s Disease: a Pragmatic Randomized Trial

Michael D. Kappelman, David A. Wohl, Hans H. Herfarth, Ann M. Firestine, Jeremy Adler, Rana F. Ammoury, Jeanine E. Aronow, Dorsey M. Bass, Julie A. Bass, Keith Benkov, …
Gastroenterology (New York, N.Y. 1943), Vol.165(1), pp.149-161.e7
07/2023
DOI: 10.1053/j.gastro.2023.03.224
PMCID: PMC10330864
PMID: 37004887

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Abstract

Tumor Necrosis Factor inhibitors (TNFi), including infliximab and adalimumab, are a mainstay of pediatric Crohn’s disease (PCD) therapy; however, non-response and loss of response is common. As combination therapy with methotrexate may improve response, we performed a multi-center, randomized, double-blind, placebo-controlled pragmatic trial to compare TNFi with oral methotrexate to TNFi monotherapy. PCD patients initiating infliximab or adalimumab were randomized in 1:1 allocation to methotrexate or placebo and followed for 12-36 months. The primary outcome was a composite indicator of treatment failure. Secondary outcomes included anti-drug antibodies (ADA) and patient reported outcomes (PROs) of pain interference and fatigue. Adverse events (AEs) and Serious AEs (SAEs) were collected. Of 297 participants (mean age 13.9 years, 35% female), 156 were assigned to methotrexate (110 infliximab initiators and 46 adalimumab initiators) and 141 to placebo (102 infliximab initiators and 39 adalimumab initiators). In the overall population, time to treatment failure did not differ by study arm (HR 0.69, 95% CI 0.45-1.05). Among infliximab initiators, there were no differences between combination and monotherapy (HR 0.93, 95% CI 0.55-1.56). Among adalimumab initiators, combination therapy was associated with longer time to treatment failure (HR 0.40, 95% CI 0.19-0.81). A trend towards lower ADA development in the combination therapy arm was not significant. [(infliximab OR 0.72 (0.49-1.07); adalimumab OR 0.71 (0.24-2.07)]. No differences in PROs were observed. Combination therapy resulted in more AEs but fewer SAEs. Among adalimumab but not infliximab initiators, PCD patients treated with methotrexate combination therapy experienced a 2-fold reduction in treatment failure with a tolerable safety profile.
adalimumab anti-tumor necrosis factor alpha children Crohn’s disease infliximab

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